Jan 23, 2023, 10:20 PM
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On 28 November, Rare Disorders NZ had a meeting with Pharmac to discuss the existing NPPA (Named Patient Pharmaceutical) policy. NPPA is Pharmac’s process for assessing whether to fund, under exceptional circumstances, a treatment that is not part of the publicly funded medicines on the Pharmaceutical Schedule for an individual patient. Only a clinician can make an application on behalf of a patient in their care, and all other funded avenues of care have had to have been explored.

Rare Disorders NZ have long held a number of concerns around the NPPA process in its current format, and voiced these concerns to the Pharmac Review panel, which subsequently included these concerns in the final Pharmac Review. These include the strict and inconsistent criteria, the lack of clarity on the principles used in the decision-making process and the narrow factors for consideration that do not factor in wider costs to families and society from not having access to treatment.

Rare Disorders NZ presented the respective concerns of the NPPA process from the perspectives of patients, clinicians, and pharmaceutical companies to Pharmac representatives at this meeting.

From the patient’s perspective, Rare Disorders NZ shared concerns around the limited interest Pharmac continues to show towards engagement with the rare disorder community and to hear lived experiences to improve their approach to delivering for the rare disorder community. The higher than necessary costs associated with not having access to an available treatment were also a major concern, including the costs of avoidable healthcare, welfare, social care, education support and disability support.

We expressed concern from the clinician’s perspective around the inefficiencies and inconsistencies of the NPPA process, including the lack of clarity around why applications have been declined and the poor awareness among clinicians about the NPPA process and how to go about it.

From the pharmaceutical companies’ perspective, we relayed concerns about the lack of initiative from Pharmac to assess new pricing offers and clinical information and of the long delays in reaching funding decisions on certain treatments.

During this presentation, Rare Disorders NZ also highlighted concerns around the infrequency in which the Rare Disorder Advisory Panel meets, and the need to include the lived experience voice in its decision-making process.

Rare Disorders NZ recommended Pharmac undertake the following steps:

  • Review of the Rare Disorders Advisory Committee membership and attendees
  • Review of the NPPA process
  • Creation of a new modern medicines policy
  • Pharmac needs to negotiate with pharmaceutical companies regarding price on treatments on the options for investment list
  • Pharmac’s engagement process needs to be reviewed given the new Pae Ora legislation and Health Quality Safety Commission code for engagement.
  • Pharmac should change its definition of a rare disorder.

Pharmac agreed to undertake the following actions from the meeting:

  • to consider how best to clarify the interface between NPPA applications and applications for Schedule listings, in order to ensure that suppliers and clinicians are not deterred from making applications;
  • to look into producing a guide/booklet for clinicians relating to the NPPA application process – and continue to consider how best to communicate outcomes back to clinicians who have made applications;
  • to endeavour to provide updated data on NPPA outcomes on Pharmac’s website within the next two months;
  • to contact RDNZ once the agenda for the next Advisory Committee meeting is confirmed to further discuss presentation of lived experience.

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